Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05398029




Registration number
NCT05398029
Ethics application status
Date submitted
19/05/2022
Date registered
31/05/2022
Date last updated
8/04/2024

Titles & IDs
Public title
A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease
Scientific title
Open-label, Phase 1b, Single-ascending Dose and Optional re Dosing Study to Evaluate the Safety of VERVE-101 Administered to Patients With Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, and Uncontrolled Hypercholesterolemia
Secondary ID [1] 0 0
VT-1001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Heterozygous Familial Hypercholesterolemia 0 0
Atherosclerotic Cardiovascular Disease 0 0
Hypercholesterolemia 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - VERVE-101

Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design - Participants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts.

Experimental: Part B: Single Dose Expansion - Participants will receive a single dose of VERVE-101 selected based on the doses studied in Part A.


Treatment: Drugs: VERVE-101
Intravenous (IV) infusion.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
Timepoint [1] 0 0
up to Day 365
Secondary outcome [1] 0 0
Evaluation of maximum observed concentration (Cmax)
Timepoint [1] 0 0
up to Day 365
Secondary outcome [2] 0 0
Evaluation of time to maximum observed concentration (tmax)
Timepoint [2] 0 0
up to Day 365
Secondary outcome [3] 0 0
Evaluation of terminal elimination half-life (t1/2)
Timepoint [3] 0 0
up to Day 365

Eligibility
Key inclusion criteria
* Male and/or female participants 18 up to 75 years at time of signing of informed consent
* Female participants not of child-bearing potential
* Diagnosis of HeFH
* Established ASCVD
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Active or history of chronic liver disease
* Current treatment with PCSK9 monoclonal antibody therapy
* Current or past treatment with inclisiran
* Clinically significant or abnormal laboratory values as defined by the protocol

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/07/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2024
Actual
Sample size
Target
44
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland
Country [2] 0 0
New Zealand
State/province [2] 0 0
Christchurch
Country [3] 0 0
United Kingdom
State/province [3] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Verve Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
VT-1001 is an open-label, phase 1b, single-ascending dose study that will evaluate the safety of VERVE-101 administered to patients with heterozygous familial hypercholesterolemia (HeFH), atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia. VERVE-101 uses base-editing technology designed to disrupt the expression of the PCSK9 gene in the liver and lower circulating PCSK9 and LDL-C in patients with established ASCVD due to HeFH. This study is designed to determine the safety and pharmacodynamic profile of VERVE-101 in this patient population.
Trial website
https://clinicaltrials.gov/study/NCT05398029
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05398029